A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome

Stopped early · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Netherton Syndrome

In brief

This study is open to people with a skin disease called Netherton syndrome (NS). People can join the study if they are 12 years or older. The purpose of this study is to find out whether a medicine called spesolimab helps people with NS. Participants are divided into a spesolimab and a placebo group. Placebo injections look like spesolimab injections but do not contain any medicine. Every participant has a 2 in 3 chance of being in the spesolimab group. In the beginning, participants get the study medicine as an injection into a vein. Afterwards, they get it as an injection under the skin every month. After 4 months, participants in the placebo group switch to spesolimab treatment. Participants are in the study for up to 3 years. During this time, they visit the study site up to 42 times. The doctors regularly check participants' NS symptoms. The results are compared between the groups to see whether spesolimab works. The doctors also regularly check participants' general health and take note of any unwanted effects.

Key facts

Study ID: NCT05856526
Run by: Boehringer Ingelheim
People needed: 43
Starts: 2023-06-12
Expected to finish: 2025-07-31
Last updated by the study team: 2026-03-12

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Male or female patients, aged 12 years and older (weight minimum is 35kg).
Confirmed diagnosis of Netherton syndrome (NS) (causative SPINK5 mutations) at baseline (Visit 2).
At least moderate severity of erythema at baseline (visit 2) (Ichthyosis Area Severity Index (IASI) score ≥ 16 and IASI-Erythema (E) score ≥8) and ≥ 3 on Investigator Global Assessment (IGA) score.
Signed and dated written informed consent and assent in accordance with International Council on Harmonisation-Good Clinical Practice (ICH-GCP) and local legislation prior to admission in the trial
Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the clinical trial protocol (CTP) as well as in the patient, parent(s) (or patient's legal guardian) information.

You may not qualify if…

Patients who have used topical corticosteroids (medium to high, US class I-V), topical retinoids, topical calcineurin inhibitors or keratolytics within 1 week prior to randomisation
Patients who have used emollient on the area to be biopsied in the previous 24 hours
Patients who have used systemic retinoids, other systemic immunosuppressants, systemic corticosteroids or phototherapy within 4 weeks prior to randomisation
Patients who have used systemic antibiotics within 2 weeks prior to randomisation
Patients who have received live vaccines within 4 weeks prior to randomisation
Patients who have received investigational products, biologics or immunoglobulins within 4 weeks or 5 half-lives (whichever is longer) prior to randomisation
Severe, progressive, or uncontrolled hepatic disease, defined as >3-fold Upper Limit of Normal (ULN) elevation in Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin
Patients who have any prior exposure to BI 655130 or another interleukin 36 receptor (IL-36R) inhibitor biologics
Further exclusion criteria apply

Where it is running

Mission Dermatology Center — Rancho Santa Margarita, California, United States
Yale University School of Medicine — New Haven, Connecticut, United States
Medical University of South Carolina — Charleston, South Carolina, United States
Virginia Clinical Research, Inc. — Norfolk, Virginia, United States
Westmead Hospital — Westmead, New South Wales, Australia
ASMC-IPSMC-skin and Veneral Diseases — Sofia, Bulgaria
Beijing Children's Hospital, Capital Medical University — Beijing, China
Southern Medical University Dermatology Hospital — Guangzhou, China
The Children's Hospital Zhejiang University School Of Medicine — Hangzhou, China
The First Affiliated Hospital, Zhejiang University — Hangzhou, China
Dermatology Hospital, Chinese Academy of Medical Sciences — Nanjing, China
Shanghai Skin Disease Hospital — Shanghai, China
Xinhua Hospital Affiliated to Shanghai Jiaotong University — Shanghai, China
HOP Saint-Louis — Paris, France
Universitätsklinikum Heidelberg — Heidelberg, Germany
Universitätsklinikum Schleswig-Holstein, Campus Kiel — Kiel, Germany
Klinikum der Universität München AÖR — München, Germany
Sourasky Medical Center — Tel Aviv, Israel
Istituto Dermopatico Dell'Immacolata - IDI - IRCCS — Roma, Italy
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino — Torino, Italy
Nagoya University Hospital — Aichi, Nagoya, Japan
Juntendo University Urayasu Hospital — Chiba, Urayasu, Japan
Okayama University Hospital — Okayama, Okayama, Japan
Hospital Tunku Azizah — Kuala Lumpur, Malaysia
Erasmus MC Sophia Kinderziekenhuis — Rotterdam, Netherlands

Full record on ClinicalTrials.gov

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